FDA requested the firm to submit a treatment protocol for expanded access.
FDA asked Protalix BioTherapeutics to submit treatment protocol related to its late-stage candidate for Gaucher’s disease to take care of the expected shortage in Genzyme’s marketed treatment. Protalix is in discussions with the agency to finalize parameters to allow patients to access prGCD.
Genzyme’s Cerezyme, a mammalian cell-expressed version of glucocerebrosidase (GCD), is the only drug approved for the treatment of Gaucher’s. The disease is a rare and serious lysosomal storage disorder. prGCD, Protalix’ lead candidate, is a plant cell-expressed recombinant form of GCD. FDA believes the development program for prGCD satisfies the regulatory criteria required to supply prGCD for expanded access to patients under a treatment protocol.
On June 16 Genzyme decided to close its manufacturing plant in Allston Landing, MA, due to viral contamination. As a result production of Cerezyme as well as Fabrazyme (for Fabry disease) was put on hold. Genzyme began shipping from finished lots of Cerezyme held in inventory, but stands to lose between $100 million and $300 million in revenues on these two drugs.